VX950 - Time to Market?

I'm confused about the possible time to market for VX950.

The FDA granted the drug Fast Track status which clears the way for clinical trials.  Right now VX950 is in a Phase II and about to open up an even larger Phase II trial for non-responders.

Realistically if SVR data holds up, how soon will the drug be made available for SOC?

CTOAN - You follow this stuff...any comments?

[desrt]

'Realistically' I wouldn't even hazard a guess until it's well into Phase 3. I believe BILN2061 was well into Phase 3 when it was yanked from human testing - and I don't think it had nearly the overt sx that VX950 does.

[bobbyullc]

here is a chart. being fast tracked i would think 2 - 3 years. hoping.

http://www.hcvadvocate.org/hepatitis/hepC/HCVDrugs_2006.pdf

[Kalio1]

There is no way it will be here in 2 or 3 years, more phase two starting in 07, then there is phase III. We dont even have SVR data on Phase I yet much less phase II! Like Dsrt mentioned, BILN2061 looked very promising but was yanked in Phase III.


It's a don't count your chickens before they hatch scenario. Even though it does look promising, there is a long way between now and availability to the public, barring  unforseen circumstance. No one can say with any certainty when or if they will be available.

[chcnme]

Hi Mike, I was told by a research coordinator that VX-950 was many years from market  (her words,  "3-4 years, if that"), and my doctor told me there were going to be "problems" with all of the newer drugs as far as drug resistance.  They both made these comments to me when we were discussing trials (I am treatment naive and wanted to wait for VX-950 because I liked the way it looked, I have heard wonderful things about it and the RVR, etc, read wonderful things, J&J partnership, fast track, had heard it would be marketed in a few years (2009)... the list goes on on the "good things".  

To my disappointment, I didn't get the feeling speaking to either of them that VX would be marketed within two years or even three, and in fact I got the impression that waiting for VX-950 might be like waiting for the moon to fall out of the sky or - at best - that I would be at least 50 years old with 35+ years of the virus before anyone ever knew if it would make it.

Here are some excerpts from an article at ClinicalCare options from :

Advances in HCV Treatment: 2006
Source: CCO Independent Conference Coverage of the 2006 Annual Meeting of the American Association for the Study of Liver Diseases*

By: Nezam H. Afdhal, MD, FRCPI, Karen L. Lindsay, MD, Mitchell L. Shiffman, MD

*CCO is an independent medical education company that provides state-of-the-art medical information to healthcare professionals through conference coverage and other educational programs.

===========================
To read it all it (and - if you want - 100s of other pages just like it on VX-950 and other newer HCV drugs and the findings in trials and what was presented at the AASLD this year, you have to register to the site, and then you can type in the search box VX-950 or ... type in whatever drug you want to read about and who said what about it.  

It gets very confusing  (or to me it does.)

http://clinicaloptions.com/layouts/CCO.Web.aspx?path=/CCO/hepatitis/conference+coverage/boston+2006/tracks/hcv+treatment/hcv+treatment/pages/page+1&layout={89AD1AF7-1174-436A-B482-E964EA6098A0}

Here is a fraction of what was presented and discussed on VX-950.  

Mitchell L. Shiffman, MD:
In a separate 28-day study by Rodriguez-Torres and colleagues,[3] 12 treatment-naive HCV genotype 1 patients were treated with telaprevir, peginterferon alfa-2a, and ribavirin for 28 days. At the end of the treatment period, all 12 patients had undetectable HCV RNA, and at 12 weeks of follow-up, 11 patients remained HCV RNA negative. Two subjects had viral variants that occurred early with dosing, but by Day 22 of treatment, both subjects were HCV RNA negative. These findings suggest that combination telaprevir treatment with peginterferon alfa-2a may prevent the development of clinically significant resistance to HCV protease inhibitors in treatment-naive patients.

Nezam H. Afdhal, MD, FRCPI:
We should note that this may not always be the case; these patients were treatment naive and were likely to be sensitive to peginterferon alfa-2a therapy itself. It is also likely that the risk of resistance would be quite different in a patient population that is known to respond poorly to peginterferon treatment or in the case of patients who were previous nonresponders.

Karen L. Lindsay, MD:
I think it is important that we clearly define the terms we are using.

[miked]

As always, thanks again for the good information; this really is THE place to get current data.

Here's what I'm pondering....I went through INF/RBV tx 18 months ago, dropped from VL of 3M to like 15,000 but started to rise around week 32 and stopped.

Biopsy after stopping tx said I'm like a .5  Then a year later a chemical marker test said I'm somewhere between a 1 and 2 which is a large difference from .5!

My Doc says wait for new drugs.  IU Medical Center says they will be in the non-responder trial early in 2007 and I would definetly qualify.  

I own a business that has a lot of travel and customer interface.  When I last treated I walked around like a zombie.

If I got in the trial and could kill the bug in 3-6 months I'd do it in a heartbeat.  If I had to go the full 12 months then I'd do it only if my liver was out of time.

So....I'm wrestling with what to believe about the conflicting biopsy v.s. chemical marker and also if I want to unnecessarily jeporize my career and income if I have the time to wait.

For the most part I'm a very decicive guy but with all this conflicting data I'm uncertain.  Any comments from you guys?

Oh BTW....Happy New Year to everyone on the Forum.  I don't know what I'd do without your inputs.

Mike

[Kalio1]

It's a tough place to be that's for sure. I would go by your biopsy results over chemical marker tests. Chemical marker tests are not all that reliable I was told.

Since you do not have much damage and have life concerns about your ability to treat coupled with a promise of being in the trial, in your shoes I'd wait. You arent having to wait until the drugs are available to the public, you will get them before that happens if you are in the phase III trials and those are coming up in the next 6 months. But then you have to hope you get into the NON placebo arm of the trial.

You seem a good candidate for a "watch and wait" approach given your limited damage and that you feel good and aren't having symptoms. I can't imagine running a business and doing a lot of travelling on tx but there are others here that have managed it.

I'd be very happy with those biopsy results, try not to stress too much over the chemical marker tests. My doc doesn't like them, he feels they are too inaccurate to use them as a definitive tool. Good luck with whatever you decide.

[Htownwife]

My husband is treating now. He also has his own business, travels, and has much customer interface. He's also an engineer and has had to design, etc. while on treatment. He has had to travel both in and out of the country while treating and has conducted trainings to large groups of people. He was extremely concerned about this going into treatment. It has slowed him down, but not stopped him in any way. Having your own business does afford you the time to stop and take breaks when you need to. I know it's different for everyone, but he's managed so far. I hope this has helped a little. Good luck with your decision!!
BTW,he was a stage 2, level 3, and has had this for 40 years.

[chcnme]

Hi Mike.  Are you saying that one doc has told you to wait for newer drugs and that another at a different facility has told you that you are a good candidate for the upcoming trial with VX (treat in 2007 in the nonresponder PROVE3?)   If so, yeah - that's a hard decision.

Here's some things I would want to know from a doc who wanted me to wait for newer drugs:  how long he thinks I can wait and how he determines when time is running out to treat me;  how accurate he believes your liver biopsy was done post treatment and what, if any significance he puts in the other test  (the chemical marker you had done a year after the biopsy).  I'd want to know, too, when another biopsy is going to be done.  The utility of liver biopsy is even becoming hotly debated, too.  Some docs  (mine included, who

[ChrisDDD]

I hear your quandary there loud and clear.  I'm very frustrated with being told that I don't have time to wait G2, S3, but now may not be able to take interferon ever again due to retinal hemorrhaging.  I'm still trying very hard to get into the VX 950 trial for non-responders.  The only other thing that's been recommended to me is low-dose "maintenance" interferon, which I also may not be able to do because of the retinal issues.

I hear you about working while on treatment.  I worked full-time the first two treatments, and it was a *****!!  I got full disability for my third treatment earlier this year and like it much better.  I was hoping the drug would work better as I could rest a lot and drink lots of water.  I don't even know if I could work now because my brain seems very foggy, to say the least.  It seems much worse to me than it was before treatment.  I have started stuttering, say stupid things that don't make any sense, and can't remember anything!!

By the way, chcnme, since you posted the results of this liver conference, my guru doc I've referred to is Dr. Ahfdal, the one quoted in different parts of the excellent post you made.

One more point to Miked, I would go by your biopsy results rather than that chemical marker.  It's interesting that biopsies may not be helpful in the future, since they SUCK!  The difficult part of this disease I've been told is that you can have high ALT and AST numbers or VL and have little liver damage, or have low numbers and lots of liver damamge.  The only way to be certain is the biopsy.  But what if they biopsy the wrong part of the liver? ...

[Imagine]

Looking on the brighter side Chris at least you don't remember the stupid things that don't make any sense that you say. I remember mine!!!!!

I am a relasper looking at trying to get the vertex Prove3 trial.

Good luck to you

[ChrisDDD]

I second the idea that Vertex enroll 10,000 patients for Prove 3, though I'm still getting the understanding that the trial I'm hoping for is a Phase II for non-responders, (Prove 2?) simply because it will be sooner than two to three years away.

Yeah, Dr. Ahfdal rocks!  He's Persian, you know.  We asked him his nationality.  Has anyone been inclined to buy stock in Vertex at this point?  I have!

To "Imagine", unfortunately for me, my wonderful family feels it is their duty to inform me of my many garbled and unintelliglbe comments, much less my frequent lapses of memory, if I even have one left!  All lovingly, though, which helps.  However, it is kind of nice sometimes to not have much of a memory, isn't it?  One could imagine many benefits to such a possibility.  Hmm ....

[SixtiesSage]

I had Dr Afdhal during my triple therapy clinical trial using Scherings PEG (It had just come out), ribaviron, and amantadine. Dr Afdhal is a very nice rather large gentleman with an English accent. A real nice guy and lots of fun to listen to!

Remember that with the FDA they can do anything they want especially if the need is great and it is! Maybe they will allow a preliminary release, say 10,000 patients as part of phase 3.

Lets hope!

[chcnme]

Hi Chris! Your doctor is Dr. Ahfdal!  That's wonderful!  I think you are in very good, if not the best hands you can be in.  I'm wishing you the best with whatever you decide  (or however they help you decide to make the decision).  It's a tough decision.  

I've often wondered, too (on the biopsy) "what if they got the wrong part of my liver; what if it really wasn't that bad;  what if what if what it" :)  When my brain starts having the 1000 question storm, I turn to a nice cup of decaf :)

Best of luck to you. Let us know how things are going for you.

[bobbyullc]

my dr says 2 years. even if longer the new data will be out in
6 mo-1 year and will say much on how well vx is working. johnson drugs just bet $170,000 million that it will be a breakthrough. they are more in the know than me. vertex went from $7 to $38 on trials. MANY people in the know are beting it WILL work. if you can wait it sure makes sense to me. 3-6months with 90% chance of svr to 48 weeks with 45% chance of svr? no brainer.

[chcnme]

Hi Chris.  It's neat to meet a patient of Dr. Ahfdal's and one who will hopefully be in the nonresponder trial!  I will be keeping my fingers, toes, legs, arms, and everything crossed that you get in and that things will be AOK with the prior retinal issues. I've only met a few people (maybe two) in the past 3 1/2 years who did experience some kind of retinal problems and had to come off tx.  That would be scary (not to mention it would take the wind right out of your sails to think that occurrence might prevent you from being treated.)  My vision has gotten pretty bad these past few years (not due to treatment).  I still haven't gotten used to my new glasses (got them a few weeks ago). They make me feel dizzy, and so far - they have been hanging around my neck more than on my eyes!

I've got a few family members who invested in VX last year because little ole "knows-nothing-about-stocks" me said one day "you might want to look at this."  I had no idea they would be interested enough to make an investment.  One has an advisor who pulled the stop loss on it because ...well - I guess because of the obvious - he thinks it'll recover.  I'll be the first to admit, I kind of felt "all important like" - my dreaded Hep C suddenly had "appeal" to someone - lol. Sick thinking, I know.  

You have a great day! Wishing you a GREAT 2007!

[copyman]

HCV without tx affects the eye sight i.e., blurred vision, floaters, and general poor vision. this is one of the known "common" symptoms with just having the disease.

[chcnme]

Yeah, I know it is :(  I got kind of worried about that, too, (when my vision started getting worse and worse this past few year and it was apparent I needed to see the eye doc.)  I was squinting at everything I tried to read.  I didn't know how bad my vision had gotten until I put those new glasses on!  LOL!  I could see EVERYTHING!   I have not had floaters, yet.  Well, maybe once or twice in the past few years -- few seconds I saw a little strange white thing floating across my field of vision, but it went away fast.  I've heard some people see them all day; that would be awful!  When I had the eye exam done last month (hadn't had one in at least 15 years)  the doc said "well, no disease process going on in there, which is good news - you have simply reached the ripe old age of presbyopia."   (I thought - whew, thank god for just presbyopia! :)

[miked]

Guys, as always thanks for the additional data points.  

Right after my diagnosis all I wanted to do was get the bug out of my body; it was like an obsession.  In business I'm very aggessive at managing situations and getting the results I want.  I went into tx bullish and somewhat naive.  After 32 weeks of side effects and failed tx, I was humbled.

This time around I'm trying to be much smarter with how I treat and what are the costs (family, business, health, etc.).

Hopefully I'll be invited to join the VX950 trial in the beginning of the year and evaluate all the issues to treating.

I know that I can come here to ask questions and get support.

Again, thanks and Happy New Year.

Mike

[PhoenixFawks]

Kalio 1, in your above referenced chicken-egg scenario, one may question whether it was a chicken or an egg.

You stated, "I believe BILN2061 was well into Phase 3 when it was yanked from human testing - and I don't think it had nearly the overt sx that VX950 does."

I would hardly consider three tests, consisting of a total of 51 patients who took the compound for a total of TWO days, a phase III clinical trial.  Did you mean phase I, or perhaps something else?

http://www.hepatitis-central.com/mt/archives/2004/12/biln_2061_for_h.html

--pf

[pln]

Hi, I was wondering how you have been... I know you watch vx, and you have helped me in the past, for that I thank you.. I have 2 more shots then I am done, 24 weeks,, wow, time flies when you are having fun. I have been UND since day 4. Will post more later. Happy new year. Pam

[couldn't think of a nickname]

Miked,

It's been quite a while since I have even checked the forum, and it looks like I missed a lot of drama. For that I am glad.

First off, BILN was in phase 1B. I don't know where that phase 3 stuff came from. They have never explicitely stated why they stopped, but it seems to be cardio-tox issues at higher doses in monkeys IIRC.

Also, phase III for 950 is expected to start WHILE phase II is ongoing, and should be the second half of next year. It seems they are still on target for a late 2008 filing, and it should only be a 6 month review time, so sometime in 2009 remains their goal. SVR 12 will be out 3 months after the last news release, from a couple of weeks ago. We should have further SVR data from the earlier trials that they reported on also (5 of 6 had SVR 12)

Since I have not been here, and I have only scanned some of the talk above, I want to throw in my 2 cents based on limited reading/understanding/knowledge of what has been going on.

First, this is the internet (I know, news flash). This creates issues that never existed before. ALL drug companies want trial information blinded, because there are too many problems when bits and pieces come out, misunderstandings, etc. Any scientist will tell you, the process must be untainted, and I imagine many people talking might be seen as possibly influencing others, or info getting out to certain segments of the population, but not all. While I have read and appreciated the updates from those on trials, I had mixed feelings from the start. I said so a long time ago. I doubt VRTX has a mole here, but then again, I don't really know the story that has been apparently ongoing here. But, I am just about 100% sure they READ the board. Oh, and when I do come in, I will still look for how everyone in the trial is doing, and everyone else that I have gotten to know in here.

Happy New Year everyone!

[miked]

Thanks for your reply and information.  

Never thought of the Mole theory but you could be right.

I'm really looking forward to reading the SVR data when it comes out.  Who cares if everyone is UND if the bug comes back.

One thing that I wanted to ask you is, "What do you think about the recent report of people with SVR still having replicating virus in their livers?"  My question is do think that a PI like VX950 will also halt the replication?

For what it's worth I always appreciate your research and am clueless why people on the board beat you up.  Keep in touch.

Mike

[PhoenixFawks]

Sigh...I misquoted desrt for Kalio 1.  The intent however, remains the same.

To err is human, to forgive, divine.

peace out --pf

[couldn't think of a nickname]

Pln, wow, day 4, that is awesome! I am sure you will attain SVR!
I hope you are recovering, from more than the illness, but soon also from the treatment.

Miked,
First, just wanted to note my absence from the forum of late has nothing to do with the forum. I've been quite busy of late, and doing some different things, so it's been partly a lack of time, and frankly, the distraction is good as I think about HCV a little less. Therefore, I find myself looking at the forum less. I'll say this, it is hard to catch up even after several days LOL. If some ask me stuff, and I don't answer, that just means I haven't been in here, but I will try to check in once in a while. It's funny, but the less I think about it, and the forum is a reminder, I think the better my mind is at least. I guess that's a little strange. Maybe not.

I guess it is an occult issue, and that is one thing that has really been on my mind. So much so, that I actually called VRTX once and asked if they would monitor for that in trials. Keep in mind, these weren't scientists I was speaking to, just pr, but I was told no. IMHO, they should, as it would be a no-lose proposition. If it doesn't, then it isn't any different than the current meds, if it does, it's a plus.

Small molecule drugs, from what I hear, and I don't know this for sure at all, are supposed to be able to deal with that issue better than current treatment. That is theoretical though until studies are done, and I wish they'd do them.

FWIW, the 5 out of 6 that got SVR12, I feel those based on current studies, should have an excellent chance for durable SVR. What I am interested in is 950 without riba in Europe.

Happy New Year and good health to all, and be well.

[prettydamscared]

I am so glad to see you posting back on this board again.  I understand you have time constraints and it sounds like it's better for you sometimes not to be so actively involved but for whatever it's worth, I'm delighted to see you back.

So, while I have you, (if you're still here) can I ask you a quick question?  I just recently found out I'm in group B, the 48 week group, but won't find out my viral loads until next week.  I am struggling with making the decision as to whether to stay on treatment as a 1A for the whole 48 weeks or stop at 24.

I've read all the studies that I can find that say achieving rvr at 4 weeks may be an indicator that it's ok to stop treating at 24 weeks even for 1a and 1b.  I should qualify that.  I'm not actually reading the studies, just the abstracts.  I'm not sure how to go about getting my hands on the actual studies and don't know that I would fully understand them anyway. I'm referring to Jensen, et al, Abstract ID 65969, Jules Levin's conference report at the AASLD Oct. 06 conference in Boston, and a retrospective study of the Hadzyiannis data done by Jensen and colleagues, the results of which are "almost identical to those obtained by Zeuzem et al".

Across the board, they all seem to say "A lower baseline HCV RNA level was the only significant and independent factor associated with SVR."  and "An RVR at week 4 of treatment is the single best predictive factor for an SVR".

So, my question to you is can you explain to me at all why having a low baseline hcv rna level matters if you clear the virus very quickly, as Pam did.  She was UD at day 4.  I'll know next week which day I was UD.  But, if I was UD early on, even though I had a high starting baseline HCV RNA of 10M and 27M one month earlier, how does that factor into it if you clear early on.  I don't know if I'm making sense.  What I'm trying to say is I don't see what significance the one has to do with the other if you clear early, you're clear.  Right?  Why would having a lower baseline hcv rna be the only significant and independent factor associated with SVR if it's cleared out early on?

I don't know if you're familiar with these sorts of issues having to do with vertex, but I thought I'd give it a shot?

Any help would be much appreciated.  Really glad to see you back.

Char

[miked]

thanks guys

[chcnme]

Hi, couldn't think.  Nice to meet you.  Always good to see someone who knows some things about VX.  If you can, I would like to ask you for some clarification because I'm trying to learn all I can before I make my decision on treating with HCV 796 slated to begin .. who knows - maybe latter part of Feb.)  

OK ... back to you said:  

"Also, phase III for 950 is expected to start WHILE phase II is ongoing, and should be the second half of next year."

Second half of next year would be somewhere around June or July, 2007.  OK.  Can I ask where are you getting this information on Phase III from ?  Do you know this to be a fact?  The reason I'm asking is I have been told by someone who I consider to be very up on the trials in VX  (that would be McHutchinson's group out of Duke)  that VX is not expected to be on the market within at least 3 to 4 years, if that.  McH hasn't told me this, but a research coordinator who is very involved in VX has told me this. She blurted it out when I was asking if I could wait ,and she said "OH! VX is not anywhere NEAR market - it'll be at least 3 - 4 years if that"  (and this was after the data was just released on .. whatever arm was just released from PhaseII.  I guess it was the group who had 12 weeks  (where 9% experienced bad reactions).  

On this Phase III you are speaking about starting while Phase II is ongoing, are you by any chance talking about PROVE3 (which is - from what I understand - still part of phase II).

You said:  It seems they are still on target for a late 2008 filing, and it should only be a 6 month review time, so sometime in 2009 remains their goal.

OK - this takes my thoughts back to a "will be marketed in 2 years."  Why, then, would that research coordinator have told me "3 - 4 years, if that?"   My note to you, frustrated sounding - is not frustrated on account of anything you have said :)  It's merely frustration from trying to figure out the timing of my treatment with what I consider to be THE best shot I would have  (VX, if I can wait).  

You said:   SVR 12 will be out 3 months after the last news release, from a couple of weeks ago. We should have further SVR data from the earlier trials that they reported on also (5 of 6 had SVR 12)

I don't really understand what you mean by this - isn't VX getting ready to release some more data on what they just released and then more data on subjects dosed for 24 weeks?  (I get lost on the arms).  And then - won't we still have to wait for more data from follow on all the subjects in Phase II, PROVE1, PROVE2, and PROVE3?   (like I said, I'm losing track on all these phases vs PROVES :)

Anyhow... I'm just trying to get my ducks in a row as best I can on the knowledge base I have on VX, which I had been following like a hawk until the research coordinator basically threw cold water on the "few years" talk.  And so, if you can or feel comfortable doing so, I would be very interested to know where you are getting your information on Phase III beginning with Phase II  (because if that is true, then that would mean filing is closer, in my opinion, perhaps, than 3 - 4 years away).  

Ok .  thank you, if you have time.  Really appreciate it.  

And best of luck to all you VX'rs out there!  I'm pulling so hard for all of you!  

[chcnme]

Hi again.  After I wrote my note to you, I went on a search trying to find more about this "PROVE3" I was talking about (I was beginning to confuse myself lol), and I found this (below) in their press release (the lastest one). I knew I had read something about PROVE3, but I haven't seen anything that I can remember about PhaseIII. That's why I asked you if perhaps you meant PROVE3 (vs. phaseIII.)

And when you said "next year",  did you mean 2007 or 2008?  

OK... thanks, and if you can still back to me I'd appreciate it.  Anything and everything I can learn on VX, I so appreciate it.  

SOURCE:  http://www.vrtx.com/Pressreleases2006/pr102706.html

EXCERPT:

Telaprevir (VX-950) is an investigational oral inhibitor of HCV protease, an enzyme essential for viral replication, and is one of the most advanced investigational agents in development that specifically targets HCV. Vertex is conducting a global Phase 2b clinical development program for telaprevir consisting of three large clinical trials that are expected to enroll approximately 1000 patients with HCV at clinical centers in the United States and Europe. Vertex completed enrollment in the 260-patient, U.S.-based PROVE 1 trial in September. The PROVE 2 trial is underway in Europe and is expected to complete enrollment by year-end with approximately 320 patients. Also in the fourth quarter, Vertex expects to initiate PROVE 3, a clinical trial of telaprevir that will enroll more than 400 treatment-experienced patients. In clinical trials, telaprevir is being dosed as 750 mg every eight hours in combination with pegylated interferon alfa-2a (Pegasys

[couldn't think of a nickname]

Hi chc,
I'll answer this one at a time. These answers are pretty much out in the public domain, so I am just rehashing what's out there for the most part.

"Second half of next year would be somewhere around June or July, 2007. OK. Can I ask where are you getting this information on Phase III from ? Do you know this to be a fact? The reason I'm asking is I have been told by someone who I consider to be very up on the trials in VX (that would be McHutchinson's group out of Duke) that VX is not expected to be on the market within at least 3 to 4 years, if that. McH hasn't told me this, but a research coordinator who is very involved in VX has told me this. She blurted it out when I was asking if I could wait ,and she said "OH! VX is not anywhere NEAR market - it'll be at least 3 - 4 years if that" (and this was after the data was just released on .. whatever arm was just released from PhaseII. I guess it was the group who had 12 weeks (where 9% experienced bad reactions). "

Second half of 2007 (it is now next year lol) BEGINS in July. It doesn't necessarily mean it starts in July. It is what their timeline has been for quite a while, and they and analysts have mentioned it often. Her "if that" comment sounds as if they are trying too hard to be cautious. Current estimates do not support that. Estimates from analysts, and the timeline PROVIDING things continue to go well. That is important.

"On this Phase III you are speaking about starting while Phase II is ongoing, are you by any chance talking about PROVE3 (which is - from what I understand - still part of phase II). "

Prove III is the non-responder portion of PHASE 2. They have stated that PHASE 3 will start WHILE PHASE 2 is ongoing. Phase 3 is the final phase before filing for FDA approval. On rare occasions, drugs will get approved after Phase 2. This is probably not a candidate. They are developing a drug with MRK (orphan drug status, no good meds, specific market) that would try for that. NOT 950 in all likelihood. I won't get too deep into FDA issues.

"OK - this takes my thoughts back to a "will be marketed in 2 years." Why, then, would that research coordinator have told me "3 - 4 years, if that?" My note to you, frustrated sounding - is not frustrated on account of anything you have said :) It's merely frustration from trying to figure out the timing of my treatment with what I consider to be THE best shot I would have (VX, if I can wait). "

I don't know who said "will be marketed in 2 years", but after approval, sometimes it takes a while to fill the pipeline especially if a drug is expected to be in demand. Can't afford to run out, for example. That shouldn't be a problem. They are manufacturing product right now, but that also gets more involved. I would prefer to say, that it should be marketed SHORTLY after approval (unless something unforseen happens).
I don't know why one would say that. It could be they just don't know. 4 years is 2011, and that seems pretty unreasonable imho (again, as long as things go as planned, and so far so good). 4 years would mean something didn't go according to plan, and no one can know that right now.

"You said: SVR 12 will be out 3 months after the last news release, from a couple of weeks ago. We should have further SVR data from the earlier trials that they reported on also (5 of 6 had SVR 12)

I don't really understand what you mean by this - isn't VX getting ready to release some more data on what they just released and then more data on subjects dosed for 24 weeks? (I get lost on the arms). And then - won't we still have to wait for more data from follow on all the subjects in Phase II, PROVE1, PROVE2, and PROVE3? (like I said, I'm losing track on all these phases vs PROVES :) "

There is a ton of data that will be released this year. The SVR12 (3 month SVR) will obviously be 3 months after they reported the last data, as they have said that's when release would be. The prelim SVR from the 6 are from earlier trials, but not part of this big one. They have always released data as soon as available.

"Anyhow... I'm just trying to get my ducks in a row as best I can on the knowledge base I have on VX, which I had been following like a hawk until the research coordinator basically threw cold water on the "few years" talk. And so, if you can or feel comfortable doing so, I would be very interested to know where you are getting your information on Phase III beginning with Phase II (because if that is true, then that would mean filing is closer, in my opinion, perhaps, than 3 - 4 years away). "

All of that information is public knowledge, meaning, in SEC filings, conference calls, analyst reports (and I read most of them) plus I have experience in this area. Phase 3 beginning during Phase 2 has been discussed often by them. I don't think they would say that unless they had the ok from the FDA. There is a lot of communication there.
I have followed this drug for about 6 years now. Pretty safe to say I don't think many knew about it 6 years ago. I found it by catching a press release on them, this was before the clinic. So, my time following it is pretty long.
Phase 3 should begin the SECOND HALF of 2007. They plan on filing for approval (based on everyone's estimates) by late 2008. Being fast tracked, those drugs normally have an answer with in 6 months of the PDUFA data (Drug Users Fee Act, which is the day they file for approval).

Thank you, and best of luck to you as well.

Happy New Year all.

[couldn't think of a nickname]

The company has talked about phase 3 starting during the end of phase 2. It has been discussed so long, it really isn't given a second thought. More than one there has said it, including Dr. John Alam, if you have heard any of his presentations. You could also look at all old press releases at www.vrtx.com
I see almost no chance this gets marketed before 2009.

[chcnme]

Hi CTON.  Thanks a lot for your time and explanation.

Ok- I guess I'm uh... lol - hard headed and stubborn and want .... more?  :)  What I am looking for is a name or names (other than personal opinions from S&P or SEC) who has stated (since the 9% adverse reactions) that PhaseIII will be launched during phase II.  I have seen predictions in earlier statements released from Vertex (generic statements), but I cannot find anything recently (other than financial analysts' opinions) that speak about Phase III launching during PhaseII.   NOT that I don

[1031 broadway]

SVR for type 1 are the only important numbers I am interested in.
What are the SVR numbers for phase 1. It would so helpful if these numbers were published bi-weekly. The fact that so much money has been invested tends to alter the way accurate numbers get published. The question for people living with this horrible question mark hanging over our heads is ' What are my chances with vx 950? ' Do I have a future ? One question please, in currant treatments what amount of SVR is considered probably permanent? My sister was treated for 1 year [not type 1] and has been UD for three years. She was told after 6mo SVR that she was cured.
ps. her side affects were brutal she is a RN and could only work 2 shifts a week. [strong flu and severe arthritis using Peg and Riba

[APKhaos]

The suggestion that FDA Phase 3 testing could start while Phase 2 tests were still in progress is something I have not been able to confirm. There just don't seem to be any sources for this??

The FDA Fast Track seems to be less than specific:        

December 9, 2005

On Thursday, Vertex Pharmaceuticals Inc. announced the Food and Drug Administration has granted its hepatitis C drug "fast track" status, which formalizes agency assistance in drug development and in-process data submissions. FDA grants fast track designation, which can speed up the review process, to therapies that address an unmet need. Vertex's VX-950 therapy, which is in mid-stage human trials, has the potential to shorten the time hepatitis C patients spend on medication, the company said.

Not sure what to make of that, other than the FDA saying that it will actually be responsive, and will assist during the development and testing phases. What is the alternative? Are non-Fast Track applications consigned to the 'ignore' pile??

[chcnme]

lmao  hahaha  

In trying to figure this out and going from one report to the next report to the trial design to the next release back to the financial report back to the end of the third quarter report to the ...BLAAAAH report, that it's probably just best to try to convince myself to repeat after me:  What will be will be, what will be will be.:)  

Best of luck! I know you all can't wait to hear something more, and soon!

[couldn't think of a nickname]

FDA fast track is about something totally different....it's apples and oranges. It is about communication, more of it, submitting data in pieces, more guidance, possibility of priority review.
You will never find what you are looking for in there. I have listened to every conference call this company has had over the last 6 years on this, and analyst reports cover this also.

Yahoo does sell some analyst reports, if one wants to spend about $30 on taking the chance it is in there (I don't remember which ones mention it). Or one could call the company and ask.
Some of the archived web calls might cover it also, so one could check them.

[chcnme]

Hi CTON.  I didn't even see your note!  Thanks again.  I'll have to say - I have not listened to the webcast presentations by the company or what their analysts have had to say on those presentations (maybe I need to, eh?) :)  Maybe tomorrow?

Thanks again!  Happy New Year!

[APKhaos]

I guess the one thing that is certain is that there will be an increasing volume of data from Prove 1 and Prove 2 [soon?] this year. The more hard data, the more likely that the progress through FDA approval will be more predictable.

I guess its understandable that nobody is going to commit beyond generalities until there is sufficient hard data to support those decisions. I guess all we have right now is some promising early indications.

[chcnme]

I couldn't wait til tomorrow ?  I had to keep looking through (it's just the way I am, I guess.)  Thanks for sending me in the right direction!  This is from July 2006.

SOURCE  
http://seekingalpha.com/article/14401


excerpt from

Vertex Pharmaceuticals Q2 2006 Earnings Conference Call Transcript (VRTX)
Vertex Pharmaceuticals Inc. (VRTX)
Q2 2006 Earnings Conference Call
July 26, 2006 5:00 PM

Andrew McDonald - Thinkequity

Okay, that was my final question is what's the design of the PROVE 3 study as you have it now?

John J. Alam
It's a little early. We are -- we're working with investigators, we will, in finalizing that protocol, they will obviously be a regulatory review as well before that protocol is started. As we always do, given all of those discussions, we'll provide more detail on the design of that study when we start the study.

Andrew McDonald - Thinkequity
Okay and then maybe finally, since treatment failure population represents a significant unmet medical need there, could you file an NDA based on the data that you generate out of PROVE 3 and PROVE 1 and 2?

John J. Alam
What I've stated previously, our primary path to NDA, to the NDA in 2008 is based on having Phase III clinical data in the package and that Phase III study would start in the middle of 2007. The key point there is that we would start the Phase III program in mid-2007 while the Phase II program, all three studies, PROVES 1-3 are ongoing. As the data evolve from those studies and become -- including the control arm data -- become available to us in 2008, there's certainly strong data and as you say, I think you're right, Andrew, in particular in the treatment failure study, strong data there would maybe sufficient to support a filing based on that set of data. At this point, projecting out the specifics of how we would -- whether or not they would be sufficient is obviously a little early."

Source:
http://seekingalpha.com/article/14401


Thanks!

[APKhaos]

Very nice get! Thanks for doing the smart digging.

[couldn't think of a nickname]

nice find. I would have never found that.